Yesterday I did the calculation that if I did one of these posts every work day it would take approximately 20 years to cover 7000 rare diseases. I am only planning to do this for 1 month. But this should underline that even then there are still close to 7000 rare diseases to describe, 99.99% of which most people in the general public will ever be aware of. I can bet that GAN would be one of those although, perhaps not based on the visibility this disease has achieved and may expand in the future.

The research on GAN is moving rapidly towards the clinic and the pieces likely still needed are validated biomarkers for the disease and potential therapeutics to clear aggregates. The research on GAN could benefit other diseases that share pathological factors which are also hallmarks of Alexander disease (AxD), amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, Parkinson’s disease, dementia with Lewy bodies, neuronal IF inclusion disease (NIFID), diabetic neuropathy, spinal muscular atrophy and some forms of Charcot-Marie-Tooth disease (CMT) such as CMT 2E .